Other Ways to Fight Blindness

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The clinical development of two promising retinal disease gene
therapies — StarGen™ for Stargardt disease and
UshStat® for Usher syndrome type 1B — has
received a major boost. Sanofi, a large international pharmaceutical company, has licensed
the treatments from Oxford BioMedica, a leading gene-therapy company.

Under the terms of the agreement, Oxford BioMedica will receive an initial $3
million payment from Sanofi, and will be eligible for future royalty and
milestone development payments. The companies will continue to work together to
move the treatments through human studies and out to the marketplace.

“We are very excited to see a major pharmaceutical company make such a strong
commitment to gene therapies for inherited retinal diseases,” says Dr. Stephen
Rose, chief research officer, Foundation Fighting Blindness. “Sanofi’s
investment demonstrates the company’s confidence in the potential for these
treatments to save vision, and at the same time, it reinforces the commitment
to getting the therapies through the clinical development process and out to
the people who need them.”

StarGen is currently in a Phase I/II clinical trial at Oregon Health &
Science University (OHSU) and the Centre Hospitalier Nationale d'Ophthalmologie
des Quinze-Vingts Institut in Paris. UshStat is also in a Phase I/II human
study at OHSU. The Foundation Fighting Blindness funded critical laboratory
studies that made these human trials possible.

The treatments work by delivering copies of normal genes to the retina,
replacing the defective, disease-causing genes. Researchers believe that a
single treatment can halt disease for several years, perhaps a lifetime.

“This licensing agreement validates the Foundation’s mission in an
unprecedented fashion,” says Dr. Rose. “Our passionate founders established the
Foundation more than 40 years ago, because essentially nothing was known about
inherited retinal degenerative diseases, and no one in the commercial sector
was investigating potential treatments. But now, thanks to the Foundation’s
sustained commitment to driving groundbreaking research, we’re on industry’s
radar screen in a big way. It is an exciting milestone for the Foundation and
the millions who need sight-saving treatments.”